Effects of 3‐Hydroxy‐3‐methylglutaric Acid on Plasma and Low‐Density Lipoprotein Cholesterol Levels in Familial Hypercholesterolemia

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Abstract
Different doses of 3-hydroxy-3-methylglutaric acid (HMG) on plasma lipids were studied in a double-blind trial in 36 patients with familial hypercholesterolemia (type IIa or hyper-.beta.-lipoproteinemia). The patients were randomly assigned to 5 groups, and each group received 1 of the following treatments: placebo, or HMG 750 mg, 1500 mg, 2250 mg or 3000 mg/day. As compared to placebo, the mean plasma cholesterol levels during the 8 wk treatment period were 11 and 13% lower in the 2250 mg and 3000 mg HMG-treated groups (P < 0.034 and < 0.021, respectively). At the same dosage levels LDL [low density lipid] cholesterol was decreased by 8%. HMG had no significant effect on plasma triglycerides as compared to placebo. Discontinuation of the medication did not result in a rebound of plasma cholesterol. There were no clinical or biologic adverse effects due to the administration of HMG, and all patients maintained excellent compliance to the medication. Because of its lack of toxicity, HMG may be useful as an adjunct to diet in the treatment of familial hypercholesterolemia.